1,854
Views
56
CrossRef citations to date
0
Altmetric
Addendum

Novel opportunities for CFTR-targeting drug development using organoids

, &
Article: e27112 | Received 19 Jul 2013, Accepted 06 Nov 2013, Published online: 11 Nov 2013

Keep up to date with the latest research on this topic with citation updates for this article.

Read on this site (4)

Sarah L. Paterson, Peter J. Barry & Alexander R. Horsley. (2020) Tezacaftor and ivacaftor for the treatment of cystic fibrosis. Expert Review of Respiratory Medicine 14:1, pages 15-30.
Read now
Massimo Conese, Elisa Beccia, Annalucia Carbone, Stefano Castellani, Sante Di Gioia, Fabiola Corti, Antonella Angiolillo & Carla Colombo. (2018) The role of stem cells in cystic fibrosis disease modeling and drug discovery. Expert Opinion on Orphan Drugs 6:12, pages 707-717.
Read now
Elena Fernandez Fernandez, Chiara De Santi, Virginia De Rose & Catherine M. Greene. (2018) CFTR dysfunction in cystic fibrosis and chronic obstructive pulmonary disease. Expert Review of Respiratory Medicine 12:6, pages 483-492.
Read now
Paul McNally & Catherine M. Greene. (2018) Cystic fibrosis: a model for precision medicine. Expert Review of Precision Medicine and Drug Development 3:2, pages 107-117.
Read now

Articles from other publishers (52)

Elena Kondratyeva, Yuliya Melyanovskaya, Anna Efremova, Mariya Krasnova, Diana Mokrousova, Nataliya Bulatenko, Nika Petrova, Alexander Polyakov, Tagui Adyan, Valeriia Kovalskaia, Tatiana Bukharova, Andrey Marakhonov, Rena Zinchenko, Elena Zhekaite, Artem Buhonin & Dmitry Goldshtein. (2023) Clinical and Genetic Characteristics of a Patient with Cystic Fibrosis with a Complex Allele [E217G;G509D] and Functional Evaluation of the CFTR Channel. Genes 14:9, pages 1705.
Crossref
Zahra Davoudi, Todd Atherly, Dana C. Borcherding, Albert E. Jergens, Michael Wannemuehler, Terrence A. Barrett & Qun Wang. (2023) Study Transportation of Drugs within Newly Established Murine Colon Organoid Systems. Advanced Biology.
Crossref
M. G. Krasnova, Yu. L. Melianovskaya, S. A. Krasovskiy, N. V. Bulatenko, A. S. Efremova, T. B. Bukharova & D. V. Goldshtein. (2023) Description of the clinical picture and assessment of functional activity of the CFTR channel in a patient with a complex allele [S466X; R1070Q]. PULMONOLOGIYA 33:2, pages 233-242.
Crossref
Elena Kondratyeva, Anna Efremova, Yuliya Melyanovskaya, Anna Voronkova, Alexander Polyakov, Nataliya Bulatenko, Tagui Adyan, Viktoriya Sherman, Valeriia Kovalskaia, Nika Petrova, Marina Starinova, Tatiana Bukharova, Sergei Kutsev & Dmitry Goldshtein. (2022) Evaluation of the Complex p.[Leu467Phe;Phe508del] CFTR Allele in the Intestinal Organoids Model: Implications for Therapy. International Journal of Molecular Sciences 23:18, pages 10377.
Crossref
Sharon L. Wong, Nikhil T. Awatade, Miro A. Astore, Katelin M. Allan, Michael J. CarnellIveta SlapetovaPo-chia Chen, Jeffry Setiadi, Elvis Pandzic, Laura K. Fawcett, John R. Widger, Renee M. Whan, Renate Griffith, Chee Y. Ooi, Serdar Kuyucak, Adam Jaffe & Shafagh A. Waters. (2022) Molecular Dynamics and Theratyping in Airway and Gut Organoids Reveal R352Q-CFTR Conductance Defect. American Journal of Respiratory Cell and Molecular Biology 67:1, pages 99-111.
Crossref
Alexander A. Baranov, Leyla S. Namazova-Baranova, Sergey I. Kutsev, Sergey N. Avdeev, Elena V. Polevichenko, Andrey S. Belevskiy, Elena I. Kondratyeva, Olga I. Simonova, Nataliya Yu. Kashirskaya, Victoria D. Sherman, Anna Yu. Voronkova, Evgeniya L. Amelina, Tatyana E. Gembitskaya, Stanislav A. Krasovskiy, Alexey G. Chermenskiy, Tatyana A. Stepanenko, Liliia R. Selimzyanova, Elena A. Vishneva, Yulia V. Gorinova, Elena A. Roslavtseva, Irina K. Asherova, Natalya A. Ilyenkova, Sergey K. Zyryanov, Niso D. Odinayeva, Tatyana Yu. Maksimycheva, Alexander V. Orlov, Sergey Yu. Semykin, Marina Yu. Chernukha, Igor A. Shaginyan, Lusine R. Avetisyan, Galina L. Shumkova, Natalya A. Krylova, Ivan A. Dronov, Maria N. Kostyleva, Ludmila A. Zhelenina, Nikolay N. Klimko, Yuliya V. Borzova, Natalya V. Vasilyeva, Tatyana S. Bogomolova, Anna A. Speranskaya, Irina A. Baranova, Evgeny G. Furman, Vera V. Shadrina, Nikolay F. Shchapov, Nika V. Petrova, Ivan V. Pashkov, Olga M. Tsirulnikova, Dmitriy P. Polyakov, Valeriy M. Svistushkin, Eduard V. Sin'kov, Vyacheslav B. Chernykh, Svetlana A. Repina, Dmitriy A. Blagovidov, Mikhail P. Kostinov, Olga V. Kondratenko, Artem V. Lyamin, Svetlana V. Polikarpova, Alexander V. Polyakov, Tagui A. Adyan, Dmitry V. Goldshtein, Tatiana B. Bukharova, Anna S. Efremova, Elena S. Ovsyankina, Ludmila V. Panova & Irina V. Cherkashina. (2022) Modern Approaches in Management of Children with Cystic fibrosis. Pediatric pharmacology 19:2, pages 153-195.
Crossref
Xiao-Yan Tang, Shanshan Wu, Da Wang, Chu Chu, Yuan Hong, Mengdan Tao, Hao Hu, Min Xu, Xing Guo & Yan Liu. (2022) Human organoids in basic research and clinical applications. Signal Transduction and Targeted Therapy 7:1.
Crossref
Ilenia Martinelli, Seyed Khosrow Tayebati, Daniele Tomassoni, Giulio Nittari, Proshanta Roy & Francesco Amenta. (2022) Brain and Retinal Organoids for Disease Modeling: The Importance of In Vitro Blood–Brain and Retinal Barriers Studies. Cells 11:7, pages 1120.
Crossref
Jeremy Sugarman. 2022. Brain Organoids in Research and Therapy. Brain Organoids in Research and Therapy 121 130 .
Meng-Meng Zhang, Ke-Lu Yang, Yan-Cheng Cui, Yu-Shi Zhou, Hao-Ran Zhang, Quan Wang, Ying-Jiang Ye, Shan Wang & Ke-Wei Jiang. (2021) Current Trends and Research Topics Regarding Intestinal Organoids: An Overview Based on Bibliometrics. Frontiers in Cell and Developmental Biology 9.
Crossref
Elena Kondratyeva, Tatyana Bukharova, Anna Efremova, Yuliya Melyanovskaya, Natalia Bulatenko, Ksenia Davydenko, Alexandra Filatova, Mikhail Skoblov, Stanislav Krasovsky, Nika Petrova, Alexander Polyakov, Tagui Adyan, Elena Amelina, Vera Shadrina, Elena Zhekaite, Aysa Zodbinova, Alexander Chernyak, Rena Zinchenko, Sergei Kutsev & Dmitry Goldshtein. (2021) Health Characteristics of Patients with Cystic Fibrosis whose Genotype Includes a Variant of the Nucleotide Sequence c.3140-16T>A and Functional Analysis of this Variant. Genes 12:6, pages 837.
Crossref
Michael A Lensink, Sarah N Boers, Vincent A M Gulmans, Karin R Jongsma & Annelien L Bredenoord. (2021) Mini-gut feelings: perspectives of people with cystic fibrosis on the ethics and governance of organoid biobanking. Personalized Medicine 18:3, pages 241-254.
Crossref
S. I. Kutsev, V. L. Izhevskaya & E. I. Kondratyeva. (2021) Targeted therapy for cystic fibrosis. PULMONOLOGIYA 31:2, pages 226-236.
Crossref
A. S. Efremova, Yu. L. Melyanovskaya, N. V. BulatenkoN. D. Odinaeva, A. V. Orlov, A. A. Pashkevich, T. A. Adyan & E. I. Kondratyeva. (2021) Description of rare alleles of the CFTR gene in cystic fibrosis using functional tests and forskolin-induced swelling assay in rectal organoids. PULMONOLOGIYA 31:2, pages 178-188.
Crossref
E. L. Amelina, A. S. Efremova, Yu. L. Melyanovskaya, N. V. Bulatenko, T. B. Bukharova, N. Yu. Kashirskaya, S. A. Krasovskiy & D. V. Goldshtein. (2021) Functional tests for assessment of residual CFTR channel activity and personalized selection of efficacious CFTR-modulators for cystic fibrosis patients with ‘mild’ and ‘severe’ genetic variants. PULMONOLOGIYA 31:2, pages 167-177.
Crossref
Alexandra McCarron, David Parsons & Martin Donnelley. (2021) Animal and Cell Culture Models for Cystic Fibrosis. The American Journal of Pathology 191:2, pages 228-242.
Crossref
Paola Lecca, Michela Lecca, Paola Melotti, Virginia Lotti, Sara Preato, Karina Kleinfelder, Alessia Farinazzo & Claudio Sorio. (2020) Computing organoids’ volume in medical images: the case study of cystic fibrosis. Computing organoids’ volume in medical images: the case study of cystic fibrosis.
E. I. Kondratyeva, N. D. Odinaeva, V. D. Sherman, A. S. Efremova, Yu. L. Melyanovskaya, N. V. Bulatenko, T. B. Bukharova & D. V. Goldshtein. (2020) A clinical case of cystic fibrosis patient with pathogenic N1303K genotype variant with assessment of the CFTR channel function by intestinal current measurement and forskolin-induced swelling in rectal organoids. Almanac of Clinical Medicine 49.
Crossref
Kristina Malsagova, Artur Kopylov, Alexander Stepanov, Tatyana Butkova, Alexandra Sinitsyna, Alexander Izotov & Anna Kaysheva. (2020) Biobanks—A Platform for Scientific and Biomedical Research. Diagnostics 10:7, pages 485.
Crossref
Insoo Hyun, J.C. Scharf-Deering & Jeantine E. Lunshof. (2020) Ethical issues related to brain organoid research. Brain Research 1732, pages 146653.
Crossref
Catherine Rang, Dominic Keating, John Wilson & Tom Kotsimbos. (2020) Re- Imagining Cystic Fibrosis Care : Next Generation Thinking . European Respiratory Journal, pages 1902443.
Crossref
Deena M.A. Gendoo. (2020) Bioinformatics and Computational Approaches for Analyzing Patient-Derived Disease Models in Cancer Research. Computational and Structural Biotechnology Journal.
Crossref
A.S. Efremova, Т.В. Bukharova, N.V. Petrova, N.Yu. Kashirskaya, Yu.L. Melyanovskaya, R.A. Zinchenko, E.I. Kondratyeva & D.V. Goldstein. (2020) Evaluation of CFTR Channel Functions and Responses to Modulators in Patients with Cystic Fibrosis who Have a Pathogenic F508del Variant in Their Genomes. Biotekhnologiya 36:4, pages 69-73.
Crossref
Sinobol Chusilp, Bo Li, Dorothy Lee, Carol Lee, Paisarn Vejchapipat & Agostino Pierro. (2019) Intestinal organoids in infants and children. Pediatric Surgery International 36:1, pages 1-10.
Crossref
M. Chioccioli, L. Feriani, J. Kotar, P. E. Bratcher & P. Cicuta. (2019) Phenotyping ciliary dynamics and coordination in response to CFTR-modulators in Cystic Fibrosis respiratory epithelial cells. Nature Communications 10:1.
Crossref
Silke van Koningsbruggen-Rietschel. (2019) Neue Therapiemodalitäten bei MukoviszidoseNovel treatment modalities for cystic fibrosis. Der Pneumologe 16:2, pages 88-97.
Crossref
Adedamola Olayanju, Lauren Jones, Karin Greco, Christopher E. Goldring & Tahera Ansari. (2018) Application of porcine gastrointestinal organoid units as a potential in vitro tool for drug discovery and development. Journal of Applied Toxicology 39:1, pages 4-15.
Crossref
Kevin DzoboArielle RoweDimakatso A. SenthebaneMousa A.M. AlMazyadiVictoria PattenM. Iqbal Parker. (2018) Three-Dimensional Organoids in Cancer Research: The Search for the Holy Grail of Preclinical Cancer Modeling. OMICS: A Journal of Integrative Biology 22:12, pages 733-748.
Crossref
Kathryn J. Crawford & Damian G. Downey. (2018) Theratyping in cystic fibrosis. Current Opinion in Pulmonary Medicine 24:6, pages 612-617.
Crossref
Karin M. de Winter-de Groot, Hettie M. Janssens, Rick T. van Uum, Johanna F. Dekkers, Gitte Berkers, Annelotte Vonk, Evelien Kruisselbrink, Hugo Oppelaar, Robert Vries, Hans Clevers, Roderick H.J. Houwen, Johanna C. Escher, Sjoerd G. Elias, Hugo R. de Jonge, Yolanda B. de Rijke, Harm A.W.M. Tiddens, Cornelis K. van der Ent & Jeffrey M. Beekman. (2018) Stratifying infants with cystic fibrosis for disease severity using intestinal organoid swelling as a biomarker of CFTR function. European Respiratory Journal 52:3, pages 1702529.
Crossref
Stefano Ponzano, Giulia Nigrelli, Laura Fregonese, Irmgard Eichler, Fabio Bertozzi, Tiziano Bandiera, Luis J.V. Galietta & Marisa Papaluca. (2018) A European regulatory perspective on cystic fibrosis: current treatments, trends in drug development and translational challenges for CFTR modulators. European Respiratory Review 27:148, pages 170124.
Crossref
Loukia Yiangou, Alexander D.B. Ross, Kim Jee Goh & Ludovic Vallier. (2018) Human Pluripotent Stem Cell-Derived Endoderm for Modeling Development and Clinical Applications. Cell Stem Cell 22:4, pages 485-499.
Crossref
Maria Perez-Lanzon, Guido Kroemer & Maria Chiara Maiuri. 2018. 49 81 .
Isha Dey & Neil A. Bradbury. 2018. Cell Volume Regulation. Cell Volume Regulation 337 381 .
Jennifer Guimbellot, Jyoti Sharma & Steven M. Rowe. (2017) Toward inclusive therapy with CFTR modulators: Progress and challenges. Pediatric Pulmonology 52:S48, pages S4-S14.
Crossref
Fleur Weeber, Salo N. Ooft, Krijn K. Dijkstra & Emile E. Voest. (2017) Tumor Organoids as a Pre-clinical Cancer Model for Drug Discovery. Cell Chemical Biology 24:9, pages 1092-1100.
Crossref
Andrew J. Hollins & Lee Parry. (2016) Long-Term Culture of Intestinal Cell Progenitors: An Overview of Their Development, Application, and Associated Technologies. Current Pathobiology Reports 4:4, pages 209-219.
Crossref
Jacquelien Noordhoek, Vincent Gulmans, Kors van der Ent & Jeffrey M. Beekman. (2016) Intestinal organoids and personalized medicine in cystic fibrosis. Current Opinion in Pulmonary Medicine 22:6, pages 610-616.
Crossref
Jeffrey M. Beekman. (2016) Individualized medicine using intestinal responses to CFTR potentiators and correctors. Pediatric Pulmonology 51:S44, pages S23-S34.
Crossref
Frank A.J.A. Bodewes, Henkjan J. Verkade & Micheal Wilschanski. (2016) Gastroenterological endpoints in drug trials for cystic fibrosis. Pediatric Pulmonology 51:S44, pages S18-S22.
Crossref
Lodewijk A.W. Vijftigschild, Gitte Berkers, Johanna F. Dekkers, Domenique D. Zomer-van Ommen, Elizabeth Matthes, Evelien Kruisselbrink, Annelotte Vonk, Chantal E. Hensen, Sabine Heida-Michel, Margot Geerdink, Hettie M. Janssens, Eduard A. van de Graaf, Inez Bronsveld, Karin M. de Winter-de Groot, Christof J. Majoor, Harry G.M. Heijerman, Hugo R. de Jonge, John W. Hanrahan, Cornelis K. van der Ent & Jeffrey M. Beekman. (2016) β 2 -Adrenergic receptor agonists activate CFTR in intestinal organoids and subjects with cystic fibrosis . European Respiratory Journal 48:3, pages 768-779.
Crossref
Johanna F. Dekkers, Ricardo A. Gogorza Gondra, Evelien Kruisselbrink, Annelotte M. Vonk, Hettie M. Janssens, Karin M. de Winter-de Groot, Cornelis K. van der Ent & Jeffrey M. Beekman. (2016) Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids. European Respiratory Journal 48:2, pages 451-458.
Crossref
Sarah N Boers, Johannes JM van Delden, Hans Clevers & Annelien L Bredenoord. (2016) Organoid biobanking: identifying the ethics. EMBO reports 17:7, pages 938-941.
Crossref
Johanna F. DekkersGitte BerkersEvelien KruisselbrinkAnnelotte VonkHugo R. de JongeHettie M. JanssensInez BronsveldEduard A. van de GraafEdward E. S. NieuwenhuisRoderick H. J. HouwenFrank P. VleggaarJohanna C. EscherYolanda B. de RijkeChristof J. MajoorHarry G. M. HeijermanKarin M. de Winter–de GrootHans CleversCornelis K. van der EntJeffrey M. Beekman. (2016) Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis. Science Translational Medicine 8:344.
Crossref
Sathidpak Nantasanti, Alain de Bruin, Jan Rothuizen, Louis C. Penning & Baukje A. Schotanus. (2016) Concise Review: Organoids Are a Powerful Tool for the Study of Liver Disease and Personalized Treatment Design in Humans and Animals. Stem Cells Translational Medicine 5:3, pages 325-330.
Crossref
Kristin Werner, Jürgen Weitz & Daniel E. Stange. (2016) Organoids as Model Systems for Gastrointestinal Diseases: Tissue Engineering Meets Genetic Engineering. Current Pathobiology Reports 4:1, pages 1-9.
Crossref
Dragana Vidović, Marianne S. Carlon, Mélanie F. da Cunha, Johanna F. Dekkers, Monika I. Hollenhorst, Marcel J. C. Bijvelds, Anabela S. Ramalho, Chris Van den Haute, Marc Ferrante, Veerle Baekelandt, Hettie M. Janssens, Kris De Boeck, Isabelle Sermet-Gaudelus, Hugo R. de Jonge, Rik Gijsbers, Jeffrey M. Beekman, Aleksander Edelman & Zeger Debyser. (2016) rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice. American Journal of Respiratory and Critical Care Medicine 193:3, pages 288-298.
Crossref
Alan S. Verkman, Aleksander Edelman, Margarida Amaral, Marcus A. Mall, Jeffrey M. Beekman, Torsten Meiners, Luis J.V. Galietta & Christine E. Bear. (2015) Finding new drugs to enhance anion secretion in cystic fibrosis: Toward suitable systems for better drug screening. Report on the pre-conference meeting to the 12th ECFS Basic Science Conference, Albufeira, 25–28 March 2015. Journal of Cystic Fibrosis 14:6, pages 700-705.
Crossref
Frank J Accurso. (2015) Cystic fibrosis, ivacaftor, and the Arg117His-CFTR mutation. The Lancet Respiratory Medicine 3:7, pages 498-499.
Crossref
Frank A.J.A. Bodewes, Henkjan J. Verkade, Jan A.J.M. Taminiau, Drucy Borowitz & Michael Wilschanski. (2015) Cystic fibrosis and the role of gastrointestinal outcome measures in the new era of therapeutic CFTR modulation. Journal of Cystic Fibrosis 14:2, pages 169-177.
Crossref
R. Dekkers, L.A.W. Vijftigschild, A.M. Vonk, E. Kruisselbrink, K.M. de Winter-de Groot, H.M. Janssens, C.K. van der Ent & J.M. Beekman. (2015) A bioassay using intestinal organoids to measure CFTR modulators in human plasma. Journal of Cystic Fibrosis 14:2, pages 178-181.
Crossref
Jeffrey M. Beekman, Isabelle Sermet-Gaudelus, Kris de Boeck, Tanja Gonska, Nico Derichs, Marcus A. Mall, Anil Mehta, Ulrich Martin, Mitch Drumm & Margarida D. Amaral. (2014) CFTR functional measurements in human models for diagnosis, prognosis and personalized therapy. Journal of Cystic Fibrosis 13:4, pages 363-372.
Crossref